What changed: Acute myeloid leukaemia

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With venetoclax in newly diagnosed unfit AML

Newly diagnosed AML, unfit for intensive induction, with venetoclax (ASCERTAIN-V)

7+3 plus midostaurin (ITD or TKD) or quizartinib (ITD only), consolidation with continued inhibitor, allogeneic transplant for most FLT3-ITD in CR1, post-transplant FLT3-inhibitor maintenance if MRD-positive. (NCCN Category 1 (midostaurin, quizartinib), ESMO-MCBS A (RATIFY))

ATRA + arsenic trioxide without chemotherapy for standard risk (cure >95%); ATRA + arsenic + idarubicin or gemtuzumab for high risk. Differentiation syndrome prophylaxis. (NCCN Category 1)

Marrow morphology, flow, karyotype/FISH, rapid FLT3/NPM1/IDH testing (results within days), NGS panel; ELN 2022 risk; fitness assessment. Menin-inhibitor and FLT3-inhibitor eligibility depends on these results. (NCCN Category 2A)

Intensive induction or venetoclax-azacitidine to remission, then allogeneic transplant as the only realistic cure; clinical trial strongly preferred; TP53-mutated disease has no effective targeted therapy after the magrolimab and eprenetapopt failures. (NCCN Category 2A (clinical trial preferred))

7+3 plus fractionated gemtuzumab ozogamicin (ALFA-0701); high-dose cytarabine consolidation; MRD-guided transplant for intermediate risk. (NCCN Category 2A)

CPX-351 induction (Study 301) then transplant in CR1; alternatives include 7+3 or venetoclax-based therapy in trials. (NCCN Category 1 (age 60-75))

Oral azacitidine (Onureg) for patients not transplanted (QUAZAR AML-001); FLT3 inhibitor maintenance after transplant in FLT3-ITD (MORPHO for MRD-positive); menin inhibitor maintenance in trials. (NCCN Category 1 (oral azacitidine))

Gilteritinib monotherapy (ADMIRAL) or gilteritinib + venetoclax/azacitidine, then transplant; quizartinib in Japan. (NCCN Category 1)

Ivosidenib or olutasidenib (IDH1), enasidenib (IDH2), often with azacitidine or venetoclax; differentiation-syndrome monitoring. (NCCN Category 2A)

Menin inhibitor: revumenib (KMT2Ar 2024; NPM1 2025) or ziftomenib (NPM1, November 2025), as a bridge to transplant; triplets with venetoclax-azacitidine in trials. (NCCN Category 2A)

Venetoclax + azacitidine (VIALE-A) or venetoclax + oral decitabine-cedazuridine (ASCERTAIN-V, 2026); ivosidenib + azacitidine if IDH1-mutated (AGILE); low-dose cytarabine + venetoclax as an alternative. Continue until progression; consider transplant in responders who become fit. (NCCN Category 1 (venetoclax + HMA), ESMO-MCBS 4 (VIALE-A))

CR 41.

Decitabine-cedazuridine + venetoclax approved 13 May 2026 (ASCERTAIN-V, CR 41.6%).

Relapsed/refractory NPM1-mutant AML

Relapsed/refractory NPM1-mutant AML

Preparative regimen for allogeneic HSCT in AML/MDS with fludarabine

Relapsed/refractory NPM1-mutated AML with no satisfactory alternative

CR 23%, ORR 33%, median OS 6.

Revumenib NPM1 label (October) and ziftomenib approval (13 November, KOMET-001).

CR+CRh 22.

A milestone in how this cancer is treated.

Approved for KMT2A-rearranged acute leukaemia (AUGMENT-101). Magrolimab (CD47) discontinued after ENHANCE trials, a setback for TP53-mutated AML.

Frontline FLT3-ITD AML (QuANTUM-First)

EU brand Inaqovi

Newly diagnosed FLT3-ITD AML with 7+3, consolidation, and maintenance

QuANTUM-First OS 31.9 vs 15.1 months, including patients to age 75.

Study 2102-HEM-101 pivotal cohort

Newly diagnosed IDH1-mutated AML with azacitidine (AGILE)

Relapsed/refractory IDH1-mutated AML

OS 24.

OS 31.

Genetics-first classification; ivosidenib-azacitidine OS HR 0.44; second IDH1 inhibitor approved.

Paediatric expansion

Paediatric patients ≥1 year

Venetoclax + azacitidine full approval in unfit AML (VIALE-A)

Full approval in AML (VIALE-A)

Onureg maintenance in AML

MDS/CMML

Newly diagnosed AML unfit for intensive chemotherapy, with venetoclax (VIALE-A)

Oral azacitidine (Onureg) maintenance after intensive induction (QUAZAR AML-001)

MDS and CMML

Same

Genomic assignment within seven days was feasible; patients treated on Beat AML sub-studies had median overall survival of 12.

OS 14.

OS 14.7 vs 9.6 months; full approval October 2020; oral azacitidine maintenance approved (QUAZAR).

Marketing application withdrawn after EMA objections on efficacy

Complete response letter for relapsed/refractory indication (QuANTUM-R)

Newly diagnosed IDH1-mutated AML unfit for intensive chemotherapy

Relapsed/refractory FLT3-ITD AML

OS 9.

ADMIRAL interim; full OS data 2019

Newly diagnosed AML in unfit patients with azacitidine/decitabine/LDAC (accelerated)

Newly diagnosed low-risk APL with tretinoin

Relapsed or refractory FLT3-mutated AML

Newly diagnosed AML in adults ≥75 or unfit for intensive chemotherapy, with low-dose cytarabine

Relapsed/refractory IDH1-mutated AML

Blastic plasmacytoid dendritic cell neoplasm, adults and children ≥2

AML with azacitidine/decitabine/LDAC in unfit patients

OS 9.

Ivosidenib (IDH1) and gilteritinib (ADMIRAL) approved; venetoclax + HMA/LDAC gets accelerated approval for unfit AML.

Re-approval at lower fractionated dose for newly diagnosed and relapsed CD33+ AML (ALFA-0701)

Adults

Single-arm AG221-C-001

Newly diagnosed therapy-related AML or AML with myelodysplasia-related changes, adults

Relapsed/refractory IDH2-mutated AML

Newly diagnosed and relapsed CD33+ AML

Newly diagnosed FLT3-mutated AML with 7+3 and consolidation; advanced systemic mastocytosis

Median OS 74.

Midostaurin (RATIFY), enasidenib, CPX-351, gemtuzumab re-approval; the first new AML drugs since 2000.

A milestone in how this cancer is treated.

3-year EFS 53.

Newly diagnosed de novo or secondary AML in adults 65 and over not candidates for standard induction

EFS 17.

Fractionated dosing with 7+3 improves EFS; re-approval 2017.

Voluntary withdrawal after confirmatory trial showed no benefit and excess deaths

Maintenance with interleukin-2 for adults with AML in first remission (exceptional circumstances)

First hypomethylating agent approved (MDS)

Myelodysplastic syndromes

First hypomethylating agent; later the AML backbone for venetoclax and IDH/menin combinations.

APL

Accelerated approval, relapsed CD33+ AML in older patients; first ADC ever approved

Relapsed/refractory APL

Relapsed CD33+ AML (withdrawn 2010)

Accelerated approval in relapsed CD33+ AML; withdrawn 2010 after SWOG S0106 toxicity.

A milestone in how this cancer is treated.

Induction of remission in APL (t(15;17)/PML-RARA)

Myeloid reconstitution after autologous bone marrow transplant; later AML post-induction, stem cell mobilisation, allogeneic transplant, graft failure and acute radiation syndrome

Adult acute myeloid leukaemia, as a component of combination induction chemotherapy

Shanghai group shows all-trans retinoic acid remissions; arsenic follows in the 1990s. First differentiation therapy.

Thomas (Seattle) reports long-term survival after HLA-matched sibling transplant; Nobel Prize 1990.

Cytarabine 7 days + daunorubicin 3 days (Yates et al.); still the intensive backbone.

A milestone in how this cancer is treated.

Remission induction and consolidation of acute non-lymphocytic leukaemias

Farber's aminopterin in childhood ALL; AML remissions follow with 6-MP and cytarabine in the 1960s.